Leber\u2019s congenital amaurosis is a dystrophy of the retina which provokes progressive blindness in affected children. It accounts for 10% to 20% of the cases of blindness in children.<\/p>\n
In October 2011, the Nantes University Hospital began a gene therapy trial which included 9 patients with Leber\u2019s congenital amaurosis. Objectives: to evaluate tolerance of a single administration of the experimental drug in child and adult patients suffering from a retinal dystrophy associated with a defect in the RPE65 gene; to evaluate the efficacy of the administration of the experimental drug in one eye and to evaluate the humoral and cellular immune response following the administration of the experimental drug. This trial is the result of research performed in the Atlantic Gene Therapies laboratories which, since 2006, have succeeded in restoring vision to puppies affected with this pathology. The vector for the drug was produced by Atlantic Bio GMP (French Blood Agency, AFM-Telethon, INSERM, and the Nantes University Hospital).<\/p>\n
A group in I-Stem (Christelle Monville) is working on the modeling of retinal pathologies of genetic origin using iPS cell lines which carry mutations, in order to identify biological markers associated with these mutations, and then to initiate screening of molecules which are\u00a0 potentially therapeutic for these diseases. The first retinal pathology to be studied is Leber\u2019s congenital amaurosis<\/p>\n
Based on their experience working on Leber\u2019s congenital amaurosis, teams from Nantes are exploring the preclinical development of a gene therapy strategy for two other pigmentary retinitis diseases associated with mutations in the PDE6 Beta and RPGRIP genes.<\/p>\n","protected":false},"excerpt":{"rendered":"
Leber\u2019s congenital amaurosis is a dystrophy of the retina which provokes progressive blindness in affected children. It accounts for 10% to 20% of the cases of blindness in children. Phase I\/II gene therapy trial utilizing the rAAV2\/4.hRPE65 vector for the treatment of retinal dystrophy associated with defects in the RPE65 gene Laboratoire de r\u00e9f\u00e9rence :\u00a0Atlantic … [Read more]<\/a><\/p>\n","protected":false},"author":3,"featured_media":0,"parent":4341,"menu_order":10,"comment_status":"closed","ping_status":"closed","template":"","meta":{"footnotes":""},"class_list":["post-4343","page","type-page","status-publish","hentry"],"_links":{"self":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/pages\/4343","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/users\/3"}],"replies":[{"embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/comments?post=4343"}],"version-history":[{"count":0,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/pages\/4343\/revisions"}],"up":[{"embeddable":true,"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/pages\/4341"}],"wp:attachment":[{"href":"https:\/\/www.institut-biotherapies.fr\/en\/wp-json\/wp\/v2\/media?parent=4343"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}